July 22, 2025

The need-to-know this morning

The Food and Drug Administration this morning rejected a viral-based treatment from Replimune Group intended for patients with advanced skin cancer — a decision that again signals a hardened stance on drug approvals from Vinay Prasad, the agency's top regulator of cell and gene therapies.

gene therapy

Sarepta pauses shipments of Duchenne drug

Sarepta Therapeutics said late yesterday that it will pause all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following a request by the Food and Drug Administration.

It's a surprising reversal — the company had initially rejected the agency's request, which was issued last Friday. "This proactive step will allow Sarepta the necessary time to respond to any requests for information and allow Sarepta and FDA to complete the Elevidys safety labeling supplement process," said CEO Doug Ingram said in a statement.

Read more from STAT's Adam Feuerstein and Jason Mast.

The Sarepta developments are not just a case of a company stumbling in its communications with the FDA, but rather an object lesson in the risks that come with lowering the barriers for evidence for new medicines, my colleague Matt Herper writes. Read his take here.

biotech

Narcolepsy drugs are the start of a race to help patients stay awake

NarcolepsyDrugs__Illustration_MollyFerguson_071725

Molly Ferguson for STAT

A handful of drugmakers are rapidly developing a new, promising class of drugs to treat narcolepsy. But the companies have ambitions to go much bigger — to offer the treatments to people with common health conditions that cause them to struggle to stay awake during the day.

The treatments mimic the effects of a neuropeptide called orexin. The orexin system not only regulates the cycle of sleeping and waking, but may also boost attentiveness, cognition, and mood. That opens up the possibility that these new drugs could benefit not only people with rare sleep disorders, but potentially also broad patient populations like those with Alzheimer's and depression.

The executives I spoke with drew parallels to the GLP-1 drug class, in the sense that this could be another rare instance in which medications initially studied for a narrow group could end up benefiting many different types of patients.

regulation

New FDA drug center head is a also critic of science mainstream

The FDA yesterday named George Tidmarsh, a longtime biotech executive who is an adjunct professor at Stanford, as its top drug regulator.

My colleagues dug into his background. He helped lead the development of several approved drugs and has served as CEO of companies La Jolla Pharmaceutical Company and Horizon Pharma.

Similar to other health agency heads in the Trump administration, he's criticized the academic establishment, writing in a LinkedIn post last year that "academic science has become riddled with fraud."

And in an opinion piece last year, he criticized former gene therapy regulator Peter Marks, saying that Marks "is not a hero of the resistance but instead has been subverting the scientific process at FDA for years."

Read more from STAT's Drew Joseph and Jonathan Wosen.

pharma

AstraZeneca to invest $50 billion in U.S. drugmaking

AstraZeneca announced yesterday that it will invest $50 billion in U.S. manufacturing and R&D by 2030, as the Trump administration threatens the pharma industry with potential tariffs and price controls.

At an event in D.C., AstraZeneca CEO Pascal Soriot — flanked by Trump administration officials and Virginia Gov. Glenn Youngkin — said that as part of the investment, the company would build a multibillion-dollar manufacturing site in Virginia. The factory will produce obesity drugs, including GLP-1s.